Jadeite Medicines Inc., (Jadeite Medicines) a clinical stage biopharmaceutical company headquartered in Tokyo, Japan (President & CEO, Eiichi Takahashi) has been conducting clinical studies of odevixibat in patients with PFIC in Japan under its strategic collaboration with Albireo Pharma Inc., a wholly-owned subsidiary of Ipsen (Euronext: IPN: ADR: IPSEY).

Jadeite Medicines completed the phase 3 trial (A4250-J005) and prepared the dossier required for the NDA for PFIC in Japan.

The NDA for this indication, based on the strategic collaboration, has been transferred to IPSEN Co., Ltd. headquartered in Tokyo, Japan (President & Representative Director: Kurt Rimkus), a wholly-owned subsidiary of Ipsen which is responsible for commercialization of the product.

Odevixibat is already approved for cholestatic pruritus in patients with Alagille syndrome (ALGS) and patients with PFIC in the United States and European countries and is marketed by Ipsen.

Through the development of odevixibat, Jadeite Medicines aspires to contribute to the treatment of patients suffering from PFIC. Jadeite Medicines commits to the health and quality of life for patients through our relentless R&D in drug development in order to fulfill patient unmet needs.

Progressive Familial Intrahepatic Cholestasis (PFIC)

PFIC is a spectrum of autosomal recessive genetic disorders in which cholestasis may lead to end-stage liver disease. PFIC is designated as an intractable disease (designation number 338) by the MHLW in Japan.

PFIC is estimated to affect between one in every 50,000 to 100,000 children born worldwide. PFIC represents 10% to 15% of cases of cholestasis in children. In Japan, estimated 100 patients suffer from PFIC.

Symptoms develop early with median age at onset of symptoms of approximately 3 months and jaundice, pale stools, and intractable pruritus become apparent at 3-4 months of age, accompanied by hepatosplenomegaly and growth failure. Currently there are no treatments except for symptomatic treatments in Japan and without conservative treatment patients will require surgical options including liver transplantation.

Odevixibat

Odevixibat is a once-daily, potent non-systemic ileal bile acid transport inhibitor (IBATi) that acts locally within the small intestine.

Odevixibat is approved and marketed by Ipsen in the United States, Europe and other territories for the treatment of cholestatic pruritus in patients with ALGS and patients with PFIC.

Odevixibat has also received orphan drug designation for the treatment of biliary atresia and primary biliary cirrhosis in the U.S. and Europe, and the Phase III BOLD trial in patients with biliary atresia is ongoing overseas.